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2013 NNPDF Session on Cyclodextrin in NPC Disease

On August 2nd, 2013 in Baltimore MD, the National Niemann-Pick Disease Foundation (NNPDF) hosted a morning session on the use of Cyclodextrin (2-HPBCD) in the treatment of NPC disease during it’s 21st Annual Family Support and Medical Conference.

This session was attended by about 220 people. In many ways, the session was a milestone for the NPC community. This session marked a true coming together of the NPC community along with our partners at the NIH, FDA, J&J, and the numerous foundations that have all contributed to the Cyclodextrin story.

Key messages from the meeting include; 1) The importance of collaboration and cooperation, 2) The need for a clear path forward for NPC patients that cannot participate in the formal studies, and 3) The daunting but surmountable regulatory requirements that make our path forward incredibly challenging.

The following is the agenda. There are links in the agenda to a video from each of the presenters. You can also “browse” the videos using the “next” and “prev” buttons to the right ——–>

Cyclodextrin therapy for NPC1 disease (Dan Ory)
What this means for NPC patients/parents; one slide that provides a overview of the process and clear places where everyone can see where they fit in (Included in Panel Session Part-I)